Chardan

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news. In this week’s note: 1) Rocket Pharmaceuticals (Buy) announced the FDA has lifted the clinical hold on its pivotal phase II trial of RP-A501 in Danon disease. The company will proceed with a dose of 3.8e13 GC/ kg, which is lower than the previous dose of 6.7e13 GC/kg but still within a range that showed an activity signal according to the company. 2) Ionis Pharmaceuticals, Inc. (unrated) announced FDA approval of Dawnzera (donidalorsen), a prophylactic ASO targeting plasma prekallikrein (PKK) in adults and pediatric patients 12 years and older with hereditary angioedema (HAE). Dawnzera will be priced at $57,462 per dose which is administered every 4 weeks until a patient stabilizes, after which it can be administered every 8 weeks. 3) Kite, a Gilead (unrated) company announced the planned acquisition of Interius BioTherapeutics (private), an in vivo CAR company for $350 mm in cash. Gilead has 2 approved CAR-T cell therapy programs, Yescarta and Tecartus, both traditional autologous ex vivo therapies. The acquisition gives Gilead access to Interius' phase I targeted lentiviral CAR-T and CAR-NK program, INT2104, for B cell malignancies. 4) Kriya Therapeutics, Inc. (pvt) disclosed it has secured $313 mm in funding. The company has a pipeline of nine gene therapies, including one expressing a fusion protein to inhibit C3 and C5 for geographic atrophy and another expressing engineered ion channels for trigeminal neuralgia. Clients can view the full note here: Chardan Research - Report. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch

The Chardan team is in Jackson Hole at SALT's Wyoming Blockchain Symposium. If you're there too, let's connect! To learn more, contact us at info@chardan.com.

Chardan will host its 9th Annual Genetic Medicines Conference on October 21, 2025, in New York City. This year's program will feature in-depth panel discussions, expert presentations, and 1x1 meetings, highlighting major themes in genetic medicines. For further information, please contact CorpAccess@Chardan.com. #ChardanGMConf2025 #CGMC2025 #GeneticMedicines

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news. In this week’s note: 1) Precigen (unrated) announced that the FDA has approved Papzimeos, an adenoviral vector (AdV)-based immunotherapy for the treatment of adults with recurrent respiratory papillomatosis (RRP). Papzimeos is a non-replicating AdV designed to express a fusion antigen composed of selected regions of HPV types 6 and 11 proteins. It is administered via 4 subQ injections over a span of 12 weeks. Papzimeos is the first FDA-approved treatment for adults with RRP. Its approval is notable as it was based on results from a single-arm, open-label pivotal study. 2) Sarepta Therapeutics (unrated) announced that it has sold $174 mm of Arrowhead Pharmaceuticals (Buy) common stock in a privately negotiated block trade. In addition, Sarepta will transfer $50 mm of ARWR stock to Arrowhead's treasury in satisfaction of half of a $100 mm milestone payment that Sarepta owes to Arrowhead. The milestone payment, part of a license agreement between the two companies in which Sarepta gained rights to some of Arrowhead's muscular disease assets, was triggered when Arrowhead reached an enrollment target and authorization for dose escalation in its Phase I/II study of ARO-DM1, an RNAi therapeutic for DM1. Clients can view the full note here: https://lnkd.in/dKfQd6JP. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch #Immunotherapy #GeneticMedicines

We are pleased to congratulate our client Vyome Therapeutics, Inc. on the successful completion of their merger with ReShape Lifesciences Inc.. This milestone reflects the vision and leadership of the entire team, and we would like to especially acknowledge Venkat Nelabhotla, CEO, and Krishna K. Gupta, Chairman, for their instrumental roles in bringing this transaction to a close. It has been a privilege to support Vyome throughout this process. #ChardanTransactions #MergersAndAcquisitions #ClientSuccess

Chardan Senior Research Analyst, James McIlree, published an industry update on crypto treasury trends. The report highlights a surge in public company crypto treasury announcements in 2025, with over 90 companies adopting or exploring digital asset strategies, 42 of which occurred this quarter alone. His report also notes a declining dominance of Bitcoin-only approaches as more companies diversify into other digital assets. Clients can view the full note here: https://lnkd.in/eZStJeGG. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch #Crypto

Our analyst, Keay Nakae, was a featured speaker at the 6th Annual RNA Therapeutics Summit in Boston. Keay joined an expert panel on “Attracting Investment & Securing Preclinical Funding for Early-Stage RNA Editing Therapeutics Candidates,” where he shared key insights on what investors look for in this rapidly evolving space. Thank you to the organizers and fellow panelists, Steve Seedhouse, Roger S. and Peter Nell, for fostering such an important conversation around the future of RNA therapeutics. #RNA #Therapeutics

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news. In this week’s note: 1) At the request of the FDA, Dr. Vinay Prasad has returned to the Agency as Director of Center for Biologics Evaluation and Research (CBER). His return comes a little over week after announcing his departure amidst a series of controversies including the hold placed on Sarepta Therapeutics' (unrated) Elevidys. 2) Alnylam Pharmaceuticals (Buy), published secondary outcomes of the HELIOS-B trial of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran attenuated increases in mean left ventricular wall thickness, mass and declines in left ventricular ejection fraction, among other positive effects. 3) This week there were 2 developments in the mRNA vaccine space: i) The Department of Health and Human Services (HHS) announced a wind down of its mRNA vaccine development activities under the Biomedical Advanced Research and Development Authority (BARDA). This includes the cancellation and de-scoping of 22 mRNA vaccine development investments worth $500 mm due to failure to "protect effectively against upper respiratory infections like COVID and flu" (per RFK Jr). ii) CureVac (unrated) announced the resolution of its patent litigation with acquirer BioNTech (unrated) related to mRNA-based COVID-19 vaccines. Under the agreement, CureVac and affiliate GSK (unrated) will receive an aggregate payment of $740 mm as well as 1% royalty on sales of COVID-19 vaccines in U.S. from 1 Jan 2025 onward. Clients can view the full note here: https://lnkd.in/dSSpmi2S. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch #GeneticMedicine #mRNA #Vaccines

Chardan is pleased to have acted as a financial advisor on Orchestra BioMed Holdings recent capital raise. Orchestra BioMed is a biomedical company accelerating high-impact technologies to patients through risk-reward sharing partnerships with leading medical device companies. Their partnership-enabled business model focuses on forging strategic collaborations with leading medical device companies to drive successful global commercialization of products they develop. To read more about this transaction visit: https://lnkd.in/ec9m2Mgg or reach out to info@chardan.com. #ChardanTransactions #Biotech

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news. In this week’s note: 1) The U.S. Department of Health and Human Services announced that Dr. Vinay Prasad has resigned from his position as Director of the FDA's Center for Biologics Evaluation and Research (CBER). His departure comes just under 3 months after he was appointed to the position and follows a series of cell and gene therapy-related controversies from the Agency in July. 2) Sarepta announced that it will resume shipments of Elevidys for ambulatory DMD patients after the FDA recommended removal of the hold it had previously imposed on the therapy. The FDA's reversal followed an investigation of safety data for Elevidys in ambulatory patients. 3) Allogene (unrated) announced that it will shift to a standard lymphodepletion regimen of fludarabine and cyclophosphamide (FC) in its pivotal ALPHA3 trial of the allogeneic CD19-directed CAR-T cell therapy cema-cel for large B cell lymphoma (LBCL). Previously, the company evaluated a more aggressive lymphodepletion regimen of FC + an anti-CD52 mAb designed to maximize expansion and persistence of its allo CAR-Ts; however, the death of a patient who underwent this regimen prompted the company to reevaluate its plan for ALPHA3. Clients can view the full note here: https://lnkd.in/emgpdK7a. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch #geneticmedicines #celltherapy #FDA